European Medicines Agency Grants Orphan Drug Designation to Cornerstone Pharmaceuticals’ CPI-613® (Devimistat) for Treatment of Patients with Advanced Unresectable Biliary Tract Cancer
May 24 2022 - 11:04AM
Cornerstone Pharmaceuticals, Inc. (“Cornerstone” or the “Company”),
a company focused on rare cancer therapeutics and formerly known as
Rafael Pharmaceuticals, today announced that the European Medicines
Agency (EMA) has granted orphan drug designation to CPI-613®
(devimistat) for the treatment of advanced unresectable biliary
tract cancer.
Also known as cholangiocarcinoma, biliary tract
cancer is an extremely rare and hard to treat cancer, affecting
just over 2,000 people in the UK annually with incidents steadily
increasing every year.
“We are on a mission to develop cancer
treatments for patients who have significant unmet clinical needs.
Biliary tract cancer is considered rare and aggressive, with a
large gap in effective treatment options,” said Sanjeev Luther,
President and CEO of Cornerstone Pharmaceuticals. “Our goal is that
this milestone will provide hope for patients and families in a
setting where the current prognosis is devastating.”
This orphan drug designation is the fourth
granted for devimistat by the EMA, following existing designations
for Burkitt’s lymphoma, pancreatic cancer and acute myeloid
leukemia (AML). The Food and Drug Administration (FDA) has granted
orphan drug designation for devimistat in seven indications in the
United States, including in biliary tract cancer.
“With strong and continued Phase 2 enrollment
rates and additional trial sites opening, the research community
remains engaged towards identifying the capabilities devimistat has
in biliary cancer,” said Dr. Vaibhav Sahai, MBBS, M.S.,
principal investigator with Cornerstone’s Phase 2 trial for
patients with biliary tract cancer in combination with gemcitabine
and cisplatin. “Devimistat is a potentially life-saving therapy,
bringing hope to patients and loved ones affected by biliary tract
cancer.”
About CPI-613® (Devimistat)CPI-613® (devimistat) is a
first-in-class clinical lead compound of Cornerstone, which targets
enzymes that are involved in cancer cell energy metabolism and are
located in the mitochondria of cancer cells. Devimistat is designed
to target the mitochondrial tricarboxylic acid (TCA) cycle, a
process essential to tumor cell multiplication and survival,
selectively in cancer cells. Devimistat substantially increases
cellular stress and the sensitivity of cancer cells to a diverse
range of chemotherapeutic agents. This synergy allows for potential
combinations of devimistat with lower doses of these generally
toxic drugs to be more effective with lower patient side effects.
Combination with devimistat represents a diverse range of
opportunities to substantially improve patients’ benefit in many
different cancers. The U.S. Food and Drug Administration (FDA) has
designated devimistat as an orphan drug for the treatment of
pancreatic cancer, acute myeloid leukemia, myelodysplastic
syndrome, peripheral T-cell lymphoma, soft tissue sarcoma,
Burkitt’s lymphoma and biliary tract cancer. The European Medicines
Agency (EMA) has granted orphan drug designation to devimistat for
pancreatic cancer, acute myeloid leukemia and Burkitt’s
lymphoma.
About Cornerstone Pharmaceuticals, Inc.Cornerstone
Pharmaceuticals (“Cornerstone”) is committed to the development of
therapies for rare cancers that have few to no treatment options
available. Cornerstone’s lead compound, CPI-613® (devimistat), is
designed to target the mitochondria of cancer cells in order to
disrupt their energy production, cutting off the fuel for disease
growth. Devimistat is undergoing multiple clinical trials and has
been granted orphan drug designation by the U.S. Food and Drug
Administration (FDA) for the following indications: Burkitt’s
lymphoma, biliary tract cancer, soft tissue sarcoma,
myelodysplastic syndrome (MDS) and peripheral T-cell lymphoma. In
addition, the European Medicines Agency (EMA) has granted orphan
drug designation to devimistat for Burkitt’s lymphoma.
With science stemming from the world’s most renowned
institutions and compassion that runs deep, Cornerstone strives to
improve the lives of people with rare cancers, inaugurating a new
era of hope. For more information, please visit
www.cornerstonepharma.com.
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###
Morrena Villanueva
cornerstone@antennagroup.com
(609) 201-3408
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